Last reviewed 2026-05-27 · How we verify

Miltefosine plus GM-CSF

Hospital Universitário Professor Edgard Santos · Phase 3 active Small molecule Under review

Miltefosine plus GM-CSF is a Antiparasitic immunotherapy combination Small molecule drug developed by Hospital Universitário Professor Edgard Santos. It is currently in Phase 3 development for Visceral leishmaniasis (kala-azar). Also known as: Impavido plus GM-CSF.

Miltefosine combined with GM-CSF works by inducing immunogenic cell death and enhancing immune activation to treat leishmaniasis.

Miltefosine, a small molecule with an unknown mechanism, is being studied in combination with GM-CSF for the treatment of Cutaneous Leishmaniasis. This combination is being investigated in a randomized and controlled trial, as seen in ClinicalTrials.gov under the study "Miltefosine and GM-CSF in Cutaneous Leishmaniasis".

Likelihood of approval

60.3% vs 58.3% industry baseline

If approved by FDA: likely 2028–2030

Steps remaining: NDA/BLA submission

Confidence: High

Why this estimate

Baseline phase 3 → approval rate +58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
Anti-infectives pathway favourability +2.0pp
Microbiological endpoints + non-inferiority designs raise approval rates above baseline.

Predicted approval windows by jurisdiction (conditional on FDA approval)

Regulator	Country	Likely year	Lag vs FDA
FDA	US	2028–2030	—
EMA	EU	2029–2031	+0.7 yr
MHRA	GB	2029–2031	+0.7 yr
Health Canada	CA	2029–2032	+0.9 yr
TGA	AU	2029–2032	+1.2 yr
PMDA	JP	2029–2032	+1.5 yr
NMPA	CN	2030–2033	+2.3 yr
MFDS	KR	2029–2032	+1.4 yr
CDSCO	IN	2029–2033	+1.8 yr
ANVISA	BR	2030–2033	+2.3 yr

Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).

Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.

At a glance

Generic name	Miltefosine plus GM-CSF
Also known as	Impavido plus GM-CSF
Sponsor	Hospital Universitário Professor Edgard Santos
Drug class	Antiparasitic immunotherapy combination
Target	Leishmania cell membrane; GM-CSF receptor
Modality	Small molecule
Therapeutic area	Infectious Disease / Parasitology
Phase	Phase 3

Mechanism of action

Miltefosine is an alkylphospholipid that disrupts cell membrane integrity and induces apoptosis in Leishmania parasites. GM-CSF (granulocyte-macrophage colony-stimulating factor) enhances innate and adaptive immune responses by promoting differentiation and activation of macrophages and dendritic cells, which work synergistically to improve parasite clearance and immune memory.

Approved indications

Visceral leishmaniasis (kala-azar)

Common side effects

Gastrointestinal disturbances (nausea, vomiting, diarrhea)
Injection site reactions
Fever
Headache

Key clinical trials

Miltefosine and GM-CSF in Cutaneous Leishmaniasis (PHASE3)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

Miltefosine plus GM-CSF CI brief — competitive landscape report
Miltefosine plus GM-CSF updates RSS · CI watch RSS
Hospital Universitário Professor Edgard Santos portfolio CI

Frequently asked questions about Miltefosine plus GM-CSF

What is Miltefosine plus GM-CSF?

Miltefosine plus GM-CSF is a Antiparasitic immunotherapy combination drug developed by Hospital Universitário Professor Edgard Santos, indicated for Visceral leishmaniasis (kala-azar).

How does Miltefosine plus GM-CSF work?

Miltefosine combined with GM-CSF works by inducing immunogenic cell death and enhancing immune activation to treat leishmaniasis.

What is Miltefosine plus GM-CSF used for?

Miltefosine plus GM-CSF is indicated for Visceral leishmaniasis (kala-azar).

Who makes Miltefosine plus GM-CSF?

Miltefosine plus GM-CSF is developed by Hospital Universitário Professor Edgard Santos (see full Hospital Universitário Professor Edgard Santos pipeline at /company/hospital-universit-rio-professor-edgard-santos).

Is Miltefosine plus GM-CSF also known as anything else?

Miltefosine plus GM-CSF is also known as Impavido plus GM-CSF.

What drug class is Miltefosine plus GM-CSF in?

Miltefosine plus GM-CSF belongs to the Antiparasitic immunotherapy combination class. See all Antiparasitic immunotherapy combination drugs at /class/antiparasitic-immunotherapy-combination.

What development phase is Miltefosine plus GM-CSF in?

Miltefosine plus GM-CSF is in Phase 3.

What are the side effects of Miltefosine plus GM-CSF?

Common side effects of Miltefosine plus GM-CSF include Gastrointestinal disturbances (nausea, vomiting, diarrhea), Injection site reactions, Fever, Headache.

What does Miltefosine plus GM-CSF target?

Miltefosine plus GM-CSF targets Leishmania cell membrane; GM-CSF receptor and is a Antiparasitic immunotherapy combination.

Drug class: All Antiparasitic immunotherapy combination drugs
Target: All drugs targeting Leishmania cell membrane; GM-CSF receptor
Manufacturer: Hospital Universitário Professor Edgard Santos — full pipeline
Therapeutic area: All drugs in Infectious Disease / Parasitology
Indication: Drugs for Visceral leishmaniasis (kala-azar)
Also known as: Impavido plus GM-CSF

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing